Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation

Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice

(2008) Yimin Hua et al.   AMERICAN JOURNAL OF HUMAN GENETICS

SMN Deficiency Causes Tissue-Specific Perturbations in the Repertoire of snRNAs and Widespread Defects in Splicing

(2008) Zhenxi Zhang et al.   CELL

Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

(2008) T. O. Gavrilina et al.   HUMAN MOLECULAR GENETICS

Genotypic Features of Lentivirus Transgenic Mice

(2008) M.-O. Sauvain et al.   JOURNAL OF VIROLOGY

Efficient Retrograde Neuronal Transduction Utilizing Self-complementary AAV1

(2008) Edmund R Hollis II et al.   MOLECULAR THERAPY

Plastin 3 Is a Protective Modifier of Autosomal Recessive Spinal Muscular Atrophy

(2008) G. E. Oprea et al.   SCIENCE

Neonatal Intraperitoneal or Intravenous Injections of Recombinant Adeno-Associated Virus Type 8 Transduce Dorsal Root Ganglia and Lower Motor Neurons

(2007) Kevin D. Foust et al.   HUMAN GENE THERAPY